KemPharm : Corporate Presentation – January 2023

Management Presentation

January 2023

Trademarks herein are held by their respective owners.

Cautionary Note Regarding Presentation Information

This presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation and can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include statements regarding the promise and potential impact of our preclinical or clinical trial data, including without limitation the timing and results of any clinical trials or readouts, the timing or results of any Investigational New Drug applications and NDA submissions, including the resubmission of the NDA for arimoclomol, communications with the FDA, the potential uses or benefits of arimoclomol, KP1077, SDX or any other product candidates for any specific disease indication or at any dosage, the potential benefits of any of KemPharm's product candidates, the success or timing of the launch or commercialization of AZSTARYS® or any other products or related sales milestones, the sufficiency of cash to fund operations, our plans or ability to seek funding, our plans with respect to our share repurchase program, and our strategic and product development objectives. These forward-looking statements are based on information currently available to KemPharm and its current plans or expectations and are subject to a number of known and unknown uncertainties, risks and other important factors that may cause our actual results, performance or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of KemPharm's Annual Report on Form 10-K for the year ended December 31, 2021, as updated by the Quarterly Report on Form 10-Q for the three months and nine months ended September 30, 2022, and KemPharm's other filings with the Securities and Exchange Commission.

While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to this presentation.

This presentation also may contain estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.

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KEMPHARM VALUE PROPOSITION

	Revenue-generating	Strong balance sheet
Innovative rare disease	assets with significant	which is expected to
company with a proven	commercial potential in	fund operations and
regulatory track record	areas of high unmet	U.S. commercial build
	need	into 2026

KemPharm: Recent Highlights

• Completion of the 4-year safety trial
• Ongoing collaborative dialogue and periodic meetings with the FDA
• Working to amass and characterize the new data generated since the CRL
• NDA refiling targeted as early as Q3 2023
• Matthew Plooster named Chairman of the Board of Directors
• Richard Pascoe appointed CEO
• Travis Mickle, Ph.D. transitioned to President
• Joshua Schafer appointed Chief Commercial Officer and EVP of Business Development
• Christopher Posner joined Board of Directors to serve as new independent director

KP1077

Arimoclomol Development

Program

Corporate	Strong
Balance Sheet
Initiatives	to Support
	Value Creation

• Phase 2 trial in IH initiated; Interim efficacy and safety data expected as early as Q3 2023
• SDX granted Orphan Drug Designation by FDA for treatment of IH
• Phase 1 cardiovascular trial data confirmed initial dosing strengths for Phase 2 trial in IH
• Net revenue of $2.9M includes revenue from arimoclomol EAP program in France
• Cash, cash equivalents and investments of $107.4M as of Sept. 30, 2022
• Available capital expected to extend cash runway into 2026

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Focused on Creating Future Value in High Value Areas with Significant Unmet Needs; Solid Financial Foundation Creates Opportunities

Strategic Focus on	ü Build a highly differentiated pipeline of development assets with multiple clinical and regulatory
milestones
Rare Diseases	ü Focus on high-value areas with significant unmet needs in rare disease with potential to
	internally commercialize
Arimoclomol: Treatment of	ü NDA-stage drug candidate being developed for the treatment of NPC
ü "Capital efficient" financial structure; potential for positive cash flow; no shareholder dilution
Niemann-Pick disease type C (NPC)
ü NDA resubmission as early as Q3 2023; potential KemPharm commercial candidate
KP1077: Treatment of	ü High-value opportunity with significant unmet need
Idiopathic Hypersomnia (IH) and	ü Initiated IH Phase 2 Trial in December 2022
Narcolepsy	ü Initiate Narcolepsy Phase 3 Trial post IH Phase 2 results
Other Product Opportunities	ü Leverage prodrug platform to internally develop product candidates with significant potential
value
	ü Business development activities focused on complimentary clinical-stage rare disease assets
AZSTARYS® License	ü Expanding launch of AZSTARYS provides ongoing revenue potential from royalties and
milestones
Strong Balance Sheet	ü Cash, cash equivalents and investments of $107.4M as of September 30, 2022
ü Strong cash position supports development plan and other opportunities
	ü Based on operating forecast, cash runway expected to extend into 2026

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